FDA Grants Fast Track Designation to Investigational Anthracycline Annamycin for STS Lung Metastases

The agent annamycin, which offers the potential to fulfill unmet medical needs of traditional anthracyclines, has been granted fast track designation for the treatment of soft tissue sarcoma lung metastases.

Fast track designation has been granted by the FDA to the next-generation anthracycline annamycin for the treatment of soft tissue sarcoma (STS) lung metastases, according to the company responsible for developing the agent, Moleculin Biotech, Inc.1

This designation will allow more frequent meetings with the FDA regarding the drug’s development plan in support for proper processing for approval; regular written communication with the agency about trial design and the use of biomarkers; and eligibility for priority review and accelerated approval in certain cases as well as a rolling review, which allows for the biologics license application to be submitted as completed sections rather than waiting until its completion.

“We are pleased to receive our second fast track designation from the FDA for annamycin. We now have potential pathways for accelerated approval in 2 indications, STS lung metastases and the treatment of relapsed or refractory acute myeloid leukemia,” Walter Klemp, chairman and CEO of Moleculin, said in a press release. “Not only does this make us eligible for accelerated approval and priority review for our NDA [new drug application] submission, but it serves as an important reminder of the unmet need in STS lung metastases. We are now focused on initiating our internally funded clinical trial in the US, possibly prior to mid-year. In addition, we recently announced a $1.5 million grant awarded in Poland for an investigator initiated clinical trial there for this indication which should start later this year.”

In December 2020, the FDA OK’d the company’s request to proceed with a phase 1b/2 clinical trial of annamycin in patients with STS that has metastasized to the lungs after frontline therapy.2

Data regarding annamycin’s activity were demonstrated from in vivo models, with consistently high antitumor activity noted in different types of lung-localized cancers. In addition, high uptake of the agent was noted in animal models, which was up to 34-fold higher than that of the primary frontline chemotherapy agent, doxorubicin.

No cardiotoxicity events were shown in the clinical data with annamycin. Additionally, the agent has potential to sidestep resistance mechanisms of treatment typically associated with traditional anthracyclines, therefore offering therapy potential for a patient population with an unmet medical need.

“Since the discovery in animal models of annamycin’s effectiveness in lung metastases, we have been moving quickly to begin a clinical trial in the US to study annamycin for this indication,” Klemp said.

Klemp noted that significant activity of Annamycin has also been reported for colorectal and tripe-negative breast cancer, with meaningful concentration levels achieved in the liver, spleen, and pancreas.

“Based on this promising preclinical data, we believe the ultimate market opportunity for annamycin could be much larger than just STS lung metastases,” Klemp noted. “For all these reasons, we are excited to be moving from the animal models to clinical study.”


1. Moleculin Receives FDA Approval of Fast Track Designation for Annamycin in the Treatment of Sarcoma Lung Metastases. News release. Moleculin Biotech, Inc. March 30, 2021. Accessed March 30, 2021. https://prn.to/39t3khB

2. Moleculin Announces FDA Permission to Begin Clinical Study of Annamycin for Sarcoma Lung Metastases. News release. Moleculin Biotech, Inc. December 17, 2020. Accessed March 30, 2021. https://prn.to/3diaKFN

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